Hemophilia B
167
24
31
95
Key Insights
Highlights
Success Rate
82% trial completion
Published Results
52 trials with published results (31%)
Research Maturity
95 completed trials (57% of total)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 39/100
12.6%
21 terminated out of 167 trials
81.9%
-4.6% vs benchmark
25%
42 trials in Phase 3/4
55%
52 of 95 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 95 completed trials
Clinical Trials (167)
Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B
Liver Biopsy Following Gene Therapy For Hemophilia
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Early Check: Expanded Screening in Newborns
BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B
An Open-label, Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic/Pharmacodynamic (PK/PD) Characteristics of SR604 Injection in Patients With Hemophilia A/B and Congenital Factor VII Deficiency
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Synovial Proliferation on Routine Ultrasound: Active or Inactive?
Lentiviral FIX Gene Therapy
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
ATHNdataset Registry